A G E N D A CIBMTR WORKING COMMITTEE FOR AUTOIMMUNE ... · Reingold SC, Cohen J. Cell-Based...

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A G E N D A CIBMTR WORKING COMMITTEE FOR AUTOIMMUNE DISEASES AND CELLULAR THERAPIES Orlando, Florida Thursday, February 23, 2017, 12:15 pm – 2:15 pm Co-Chair: Ian Lewis, MBBS, PhD, Royal Adelaide Hospital, Adelaide, Australia;

Telephone: +61 8 8222 3328; E-mail: [email protected] Co-Chair: David McKenna, MD, University of Minnesota Medical Center, Minneapolis, MN;

Telephone: 612-624-5736; E-mail: [email protected] Co-Chair: Stefanie Sarantopoulos, MD, PhD, Duke University Medical Center, Durham, NC;

Telephone: 919-668-4383; E-mail: [email protected] Scientific Director: Marcelo C. Pasquini, MD, MS, CIBMTR Statistical Center, Milwaukee, WI;

Telephone: 414-805-0700; E-mail: [email protected] Statistical Director: Ruta Brazauskas, PhD, CIBMTR Statistical Center, Milwaukee, WI;

Telephone: 414-456-8687; E-mail: [email protected] Statistician: Khalid Bo-Subait, MPH, CIBMTR Statistical Center, Milwaukee, WI;

Telephone: 414-805-0711; E-mail: [email protected] 1. Introduction a. Minutes and Overview Plan from February 2016 meeting (Attachment 1)

b. Introduction of incoming Co-Chair: Sara Nikiforow, MD; Dana Farber Cancer Institute; Telephone: 1-617-632-6640; E-mail: [email protected]. Thank you to Ian Lewis for all the input to this committee.

2. a.a Accrual summary (Attachment 2) a.b 3. a.c Cellular Therapy Registry Update (M Pasquini)

a.d

4. a.e Presentations, published or submitted papers

a. AD09-01 Muraro P, Pasquini M, Atkins H, Bowen J, Farge D, Fassas A, Freedman M, Georges G, Gualandi F, Hamerschlak N, Havrdova E, Kimiskidis V, Kozak T, Macardi GL, Massacesi L, Moraes D, Nash RA, Pavletic S, Ouyang J, Rovira M, Saiz A, Simoes B, Trněný M, Zhu L, Badoglio M, Zhong X, Sormani MP, Saccardi R. Long Term Outcomes after Autologous Hematopoietic Stem Cell Transplantation for Multiple Sclerosis. Published

b. Meeting Report (Cell Therapies in Multiple Sclerosis, Portugal 2015): Scolding NJ, Pasquini M, Reingold SC, Cohen J. Cell-Based Therapeutics Strategies for Multiple Sclerosis. Submitted

5. Studies in progress (Attachment 3) a. CT10-01 Donor Leukocyte Infusion versus Second Allogeneic Hematopoietic Stem Cell Transplantation

for Disease Relapse after First Allogeneic Stem Cell Transplantation (N Frey/A Loren/D Porter) Manuscript Preparation.

B. CT13-01 Utility of Unmanipulated Donor Lymphocyte Infusion (DLI) for the Treatment of Infections in Allogeneic Hematopoietic Cell Transplantation Recipients (G Akpek) Protocol Development

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Not for publication or presentation

C. AC14-01 Long Term Outcomes after Autologous Hematopoietic Cell Transplantation for Rapidly Progressive Systemic Scleroderma (D Farge) Data collection

D. AC16-01 Pattern of use and outcomes with donor lymphocyte infusion (DLI) after HLA-haploidentical allogeneic hematopoietic stem cell transplant(V Roy) Protocol Development

6. Future/proposed studies

a. PROP 1612-12 CD-19 Chimeric Antigen Receptor T Cells with or without Hematopoietic Cell Transplantation for Treatment of Refractory Acute Lymphocytic Leukemia (J Park, M. Perales, S. Nikiforow)

7. Discussions: Next Steps on ACWC Activities for 2017/2018

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Not for publication or presentation Attachment 1

MINUTES AND OVERVIEW PLAN CIBMTR WORKING COMMITTEE FOR AUTOIMMUNE DISEASES AND CELLULAR THERAPIES Honolulu, HI Thursday, February 18, 2016, 2:45 – 4:45 pm

Co-Chair: Mitchell Cairo, MD, New York Medical College, Valhalla, NY; Telephone: 914-594-3650; E-mail: [email protected]

Co-Chair: Ian Lewis, MBBS, PhD, Royal Adelaide Hospital, Adelaide, Australia; Telephone: +61 8 8222 3328; E-mail: [email protected]

Co-Chair: David McKenna, MD, University of Minnesota Medical Center, Minneapolis, MN; Telephone: 612-624-5736; E-mail: [email protected]

Co-Chair: Stefanie Sarantopoulos, MD, PhD, Duke University Medical Center, Durham, NC; Telephone: 919-668-4383; E-mail: [email protected]

Scientific Director: Marcelo C. Pasquini, MD, MS, CIBMTR Statistical Center, Milwaukee, WI; Telephone: 414-805-0700; E-mail: [email protected]

Statistical Director:

Ruta Brazauskas, PhD, CIBMTR Statistical Center, Milwaukee, WI; Telephone: 414-456-8687; E-mail: [email protected]

Statistician: Khalid Bo-Subait, MPH, CIBMTR Statistical Center, Milwaukee, WI; Telephone: 414-805-0711; E-mail: [email protected]

1. Introduction

The meeting started at 2:45pm. The Committee chairs (David McKenna, MD, Ian Lewis, MD, Mitchell Cairo, MD and Stefanie Sarantopoulos, MD) and the Scientific Director (Marcelo Pasquini, MD) welcomed the committee and started the meeting. After the brief introduction, Dr. Cairo acknowledged the contribution by the outgoing chair Mitchell Cairo, MD. The minutes from the 2015 meeting in San Diego were then approved by the committee. Ian Lewis then went over the committee mission statement

2. Presentations, Published or submitted Papers Marcelo explained that AD0901 was in the process of resubmitting the manuscript and the goal is to have the study published by June 2016.

3. Studies in progress Marcelo thanked all the committee members for their participation in the research of Autoimmune Disease and Cellular Therapy Working Committee.

CT10-01: Donor leukocyte Infusion versus Second Allogeneic Hematopoietic Stem Cell Transplantation for Disease Relapses after First Allogeneic Stem Cell Transplantation (N Frey/A Loren/D Porter) The study proposes to compare outcomes among patients with relapsed hematologic malignancies after allogeneic hematopoietic stem cell transplant (HCT) who receive

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either a second allogeneic HCT, donor leukocyte infusion (DLI) or no intervention. Factors associated with overall survival for DLI and second HCT are also of interest. Currently the draft manuscript is being finalized and the goal is to have the paper published by June of 2016. CT13-01: Utility of Donor Leukocyte Infusion (DLI) for the Treatment of Drug-resistant Viral or Fungal Infections in Allogeneic HCT Recipients: A CIBMTR Analysis (G Akpek) The study is complicated in that it requires supplemental data. There have been around 160 patients identified for this study and we have invited PI’s from two major contributing centers Memorial Sloan Kettering and Baylor to participate in this study. The supplemental data forms have been completed and the idea is to pre-populate the supplemental data forms that are going to be sent out to the centers with the patient related data that we currently have while leaving empty cells for the additional data we are asking them to complete. The goal is to be in data collection by Spring of 2016.

AC14-01: Long Term Outcomes after Autologous Hematopoietic Cell Transplantation for Rapidly Progressive Systemic Scleroderma (D Farge) This study is being done in collaboration with the Autoimmune Working Party from the EBMT. The objective of this study is to evaluate long term outcomes from autologous hematopoietic cell transplantation (HCT) for rapidly progressive systemic sclerosis (SSc) from time of transplant to three and five years after transplantation. Protocol is now under revision. The goal of the study is to finalize the protocol and start data collection by July 2016.

4. Accrual Summary

Dr. Pasquini introduced the overview plan for the working committee and the portfolio of CIBMTR data available for potential studies. There are total of 51 first allogeneic transplants and 211 first autologous transplants for autoimmune disorders registered to the CIBMTR in 2007-2015. The majority of reports are from US. The main challenge remains collection of disease related information as most data in the CIBMTR is related to transplant related variables. Autoimmune disease specific data related to prior therapy, organ function and response to transplant is often not reported. Studies underway required supplemental data on disease information to be collected from centers. There are 808 cellular therapies infusions for 606 patients submitted to CIBMTR between 2002 and 2015.

5. Future/proposed studies

PROP 1511-40 Pattern of use and outcomes with donor lymphocyte infusion after HLA- haploidentical allogeneic hematopoitic stem cell transplant (E Gupta) The proposal was presented by Vivek Roy to the working committee. The proposal aimed to evaluate the pattern and use with DLI after HLA-haploidentical allogeneic transplant. The proposal has 146 cases on the TED level track and 39 of the CRF track. Marcelo pointed out that since there is such a low number of patients with CRF level data it would be advantageous to determine which centers are major contributors to determine is some centers are more active than others. Additionally because of the limitations with TED level data supplemental data may be required. The voting results for the study were the higher of the two proposals presented and the study will be accepted into the working committee study list.

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PROP 1511-43 Factors effecting donor lymphocyte infusion success and risk for GVHD in patients with Multiple Myeloma after allogeneic hematopoietic stem cell transplantation (L Schacter) The proposal was presented by Levanto Schacter. The primary aim of the study is to evaluate the efficacy of DLI and frequency and severity of GVHD overall and by characteristics. The secondary aim is to establish specific characteristics for favorable donor/graft attributes that could be extrapolated to the initial transplant. The proposal population generated 203 TED level patients and 32 CRF level patients that received a DLI for relapse. There was a question from the committee members asking if there is a way to calculate the time interval from when a DLI occurs post-transplant. Dr. Pasquini explained that the forms to provide us with dates of infusion. This study is not going to be accepted, based on the working committee voting results and leadership recommendation.

6. CIBMTR Cellular Therapy Task Force update

Marcelo provided an update on the cellular therapy task force objective which is to build upon the existing infrastructure to develop a cellular therapy registry for research purposes. As well as develop a cost effective tool for follow up for cell therapy trials. Marcelo then touched up on the outline of the registry which will change the existing form 4000 previously known as CTRM (Cellular Therapy for Regenerative Medicine) to CTED (Cellular Therapy Essential Data Form). The model for the cellular therapy registry will allow for a patient to receive a unique ID for their pre-CTED form which collects demographic, indications, disease status prior to CT and therapy prior to CT. The cellular therapy infusion form will collect description of the product, information on manufacturing, product analysis and infusion details. The post-CTED will collect follow up infusion, recipient survival and disease status, cause of death, development of new malignancies, persistence of the product, and development of CRS. Marcelo went over some expected difference in follow up that will need to be addressed in the cellular therapy registry. This will be based on the indication but mostly on the type of product infused. For example, genetic modified cells recipients are required to be followed for 15 years by the FDA. He then discussed the different scenarios for form submission related to overlap of cellular therapy in relation to subsequent cellular therapies and HCT. Some examples were shared with committee. Also, the infusion form had to be substantially changed from what is currently used in HCT. There is a more focus on manufacturing details as this could influence outcome and interpretation of analyses. Thus a variety of fields were created to specify the product. Lastly, it is recognized that this can be a burden for centers. This initiative remains voluntary, but it will be reimbursed. Also, the ultimate goal is to standardize cellular therapy data collection to avoid each company, region or regulatory agency to create separate ones. This will facilitate data collection and analysis of aggregate data in order to inform and advance the field. The status of the registry is that the CTED level forms are completed and that they are planned on being released in the summer of 2016. The next steps include harmonizing with EBMT and other registries. Marcelo then provided an update on the status on Autoimmune Disease and mentioned that unfortunately no proposals received this year related to this subject. This working committee is established to focus on cellular therapy and autoimmune diseases. Transplants for autoimmune disease are indeed an infrequent indication, but in the last 2 years there were important trials that were completed and published demonstrating the benefit of transplantations in controlling an autoimmune process, mainly in multiple sclerosis and scleroderma. These

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transplants are being done and reported to the CIBMTR and it is important to encourage investigators to propose studies in this area to assist in answering important questions related to best regimens, patient selection, timing of transplantation and other important supportive care issues in this field. It is recognized the need for collaboration with disease specialists in order to answer these questions. There were no specific action items for this discussion. The committee leadership will continue its collaboration with EBMT for the scleroderma study and investigate ways to expand the portfolio in studies on autoimmune diseases either with transplant or cellular therapy in the next year. The meeting adjourned at 3:45 PM.

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Working Committee Overview Plan for 2016-2017


a. AD09-01 Long Term Follow-up Analysis of Patients with MS after Auto Hematopoietic cell Transplantation. This study will be published by June 2016.

b. CT10-01 Donor Leukocyte Infusion versus Second Allogeneic Hematopoietic Stem Cell

Transplantation for Disease Relapse after First Allogeneic Stem Cell Transplantation. We expect to finalize manuscript and submit for peer-review by June 2016.

c. CT13-01 Utility of Donor Leukocyte Infusion (DLI) for the Treatment of Drug-resistant Viral or Fungal

Infections in Allogeneic HCT Recipients: A CIBMTR Analysis. The supplemental data forms are finalized. We expect to in data collection June 2016.

d. AC14-01 Long Term Outcomes after Autologous Hematopoietic Cell Transplantation for Rapidly

Progressive Systemic Scleroderma. The proposal is in revision. Plan will be to finalize protocol development by June 2016.

e. AC16-01 Pattern of use and outcomes with donor lymphocyte infusion after HLA-haploidentical

allogeneic hematopoietic stem cell transplant. The goal of this study is to be starting protocol development by June 2016.

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Oversight Assignments for Working Committee Leadership (March 2015)


Ian Lewis CT10-01 DLI vs Second Allo HCT for relapse AC16-01 DLI After HLA-haploidentical allogeneic transplant

David McKenna CT13-01 DLI for viral or fungal Infections in Allo HCT

Marcelo Pasquini AD09-01 Long term follow-up with MS after auto HCT

Stephanie Sarantopoulos AC14-01 Long Term OC after auto HCT for Systemic Scleroderma

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Accrual Summary for the Autoimmune Disorders and Cellular Therapy Working Committee

Recipients of first transplant for Autoimmune Disorders registered to the CIBMTR, 2007-2016

Variable Allogeneic Autologous

Number of patients 67 398

Number of centers 37 511

Age at transplant, years Median (range) 15 (<1-61) 43 (4-71)

18-30 11 (16) 42 (11) 30-40 1 (1) 94 (24) 40-50 8 (12) 128 (32)

50-60 3 (4) 89 (22) >60 2 (3) 25 (6) Region

US 57 (85) 118 (30) Canada 3 (4) 17 (4) Europe 5 (7) 38 (10)

Asia 0 17 (4) Australia/New Zealand 1 (1) 5 (1) Mideast/Africa 0 6 (2)

Central/South America 1 (1) 197 (49) Gender Male 38 (57) 152 (38)

Female 29 (43) 246 (62) Karnofsky Score

>90 22 (33) 71 (18) <90 36 (54) 310 (78) Missing 9 (13) 17 (4)

Graft type BM 31 (46) 2 (<1) PB 25 (37) 388 (97)

UCB 11 (16) 2 (<1) Missing 0 6 (2) Donor type

Autologous HSCT 0 238 (60) HLA-identical sibling(may include non-monozygotic twin) 17 (25) 0 Sysgeneic (monozygotic twin) 1 (1) 0

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Unrelated donor 41 (61) 0 HLA-matched other relative 1 (1) 0 HLA-mismatched relative 2 (3) 0

Missing 5 (7) 160 (40) Disease Myasthenia gravis 0 2 (<1)

Multiple sclerosis 3 (4) 251 (63) Rheumatoid arthritis 0 1 (<1) Psoriatic arthritis or psoriasis 0 1 (<1)

Systemic lupus erythematosis (SLE) 4 (6) 10 (3) Polymyositis-dermatomyositis 0 1 (<1) System Scleroderma 12 (18) 80 (20)

Antiphospholipid syndrome 1 (1) 3 (<1) Other Connective tissue disease2 2 (3) 1 (<1) Churg-Strauss vasculitis 0 1 (<1)

Juvenile Idiopathic Arthritis 2 (3) 0 Other neurological disorder3 2 (3) 24 (6) ITP- Idiopathic thrombocytopenic purpura 2 (3) 1 (<1)

Hemolytic anemia 5 (7) 0 Evan syndrome 5 (7) 0 Other autoimmune cytopenia4 10 (15) 0

Crohn’s disease 6 (9) 21 (5) Other bowel disorder5 13 (19) 1 (<1)

Conditioning regimen TBI+FLUD+-others 13 (19) 0 TBI+CY+-others 2 (3) 30 (8)

TBI+ATG+-others 0 1 (<1) BU+CY+-others 8 (12) 2 (<1) BU+FLUD+-others 13 (19) 0

CY+FLUD+-others 5 (7) 20 (5) CY+ATG+-others 3 (4) 93 (23) TLI+-others 2 (3) 0

CY+-others 0 160 (40) LPAM+-others 18 (27) 48 (12) FLUD+-others 3 (4) 1 (<1)

ATG+-others 0 1 (<1)

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Others 0 42 (11) GVHD prophylaxis No GvHD Prophylaxis 1 (1) 0

TDEPLETION +- other 3 (4) 0 CD34 select alone 7 (10) 0 Cyclophosphamide +- others 3 (4) 0

FK506 + MMF +- others 12 (18) 0 FK506 + MTX +- others(not MMF) 8 (12) 0 FK506 +- others(not MMF,MTX) 4 (6) 0

FK506 alone 1 (1) 0 CSA + MMF +- others(not FK506) 13 (19) 0 CSA + MTX +- others(not MMF,FK506) 5 (7) 0

CSA +- others(not FK506,MMF,MTX) 6 (9) 0 Other GVHD Prophylaxis 3 (4) 0 Identical twin donor 1 (1) 0

Not Applicable for autologous 0 398 Time from diagnosis to transplant, months

Median (range) 29 (<1-565) 22 (<1-393)

<12 23 (34) 160 (40) 12-24 9 (13) 45 (11) 24-36 7 (10) 25 (6)

>36 28 (42) 168 (42) Year of transplant

2007-2008 10 (15) 36 (9) 2009-2010 12 (18) 65 (16) 2011-2012 12 (18) 37 (9)

2013-2014 20 (30) 48 (12) 2015-20166 13 (19) 212 (53) Median follow-up of survivors (range), months 25 (3-103) 25 (<1-100) 1Centers from us (n=24); Canada (n=4); Europe (n=9); Asia (n=2); Australia/New Zealand (n=2); Mideast/Africa (n=3); Central/South America (n=7) 2 CREST (n=1); Mixed Connective Tissue Disease (n=1); Relapsing Polychodritis (n=1) 3 Neuromyelitis optica (n=6); Stiff person syndrome (n=9); Chronic inflammatory demyelinating polyneuropathy (n=2; Immune mediated encephalopathy (n=2); Adrenomyeloneuropathy (n=1); Chronic Inflamatory polyradiculoneuropathy (n=2); Chronic-Recurring Myelitis (n=1); CIDP (n=1); Demyelinisation Neuropathy (n=1); Limbic Encephalitis (n=1); Rasmussen Encephalitis (n=1). Mediated Encephalopathy (n=2); Limbic Encephalitis (n=1); Neuromyelitis Optica (n=6); Rasmussens Encephalitis (n=1); Stiff Person Syndrome (n=5) 4 Autoimmune Neutropenia (n=2); Autoimmune Lymphoproliferative Syndrome (n=6); Autoimmune Neutropenia (n=1); Congenital neutropenia VPS45 Mutation (n=1); Erythropoetic Protoporphyria (n=1); Unidentified autoimmune disorder (n=1) 5 Ipex Syndrome (n=7); Autoimmune Enteropathy (n=2); IL-10 Receptor Mutation (n=1); IL-10 Receptor Defect; Inflammatory Bowel Disease (n=2); 6 Cases continue to be registered to CIBMTR for 2015

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Cellular Therapies- number of complete forms submitted to CIBMTR since implementation of CTRM data collection form (Form 4000) in January 2011 – June 2016. This table does not include data from the new CTED forms released July 2016.

Characteristics N (%) Number of infusions 698 Number of Patients 554 Number of centers reporting 27 Year of infusion

2002 5 (<1) 2003 5 (<1) 2004 3 (<1) 2005 7 (<1) 2006 8 (1) 2007 16 (3) 2008 41 ( 7) 2009 99 (18) 2010 61 (11) 2011 83 (15) 2012 64 (12) 2013 63 (11) 2014 46 (8) 2015 35 (6) 2016* 17 (3)

Missing 1 (<1) Indication Neurologic

Cerebral Palsy 225 (41) Congenital Hydrocephalus 56 (10)

Autism 3 (<1) Developmental Delay 17 (3) HIE 13 (2) Brain Injury 13 (1) Periventricular leukomalacia; encephalomalacia 10 (2) Hemiparesis 12 (1) Hydrocephalus 4 (<1) Apraxia 1 (<1) ACVI 1 (<1) Encephalitis 1 (<1) Infantile Neuroaxonal Dystrophy 1 (<1) Spastic Quadriplegia 1 (<1) Partial agenesis of corpus callosum 1 (<1) Perinatal hypoxic ischemic event 1 (<1)

Cardio and peripheral vascular Chronic coronary artery disease 45 (8) Acute myocardial infarction 33 (6) Claudication 6 (1)

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Characteristics N (%) Heart Failure 7 (1) Limb Ischemia 1 (<1) Thromboangitis obliterans 1 (<1)

Autoimmune disease Crohns disease 6 (<1) Diabetes mellitus type I 1 (<1)

Musculoskeletal disease Avascular necrosis of femoral head 1 (<1)

Other ALL 36 (6) NHL 9 (2) AML 7 (1) Recurrent Medulloblastoma 4 (<1) Renal transplant tolerance 6 (<1) Induce tolerance in setting of kidney transplant 7 (<1) Non-Hodgkin Lymphoma - DLBCL 2 (<1) Malignant desmoplastic small round cell tumor 1 (<1) PNET pineo neuroectodermal tumor of brain 1 (<1) Recurrent Glioblastoma 1 (<1) Relapse/progression of disease 1 (<1) Thalassemia major 1 (<1) Ischemic 2 (2) Acute biphenotypic leukemia 1 (<1) Acute kidney injury following cardiac surgery 2 (2)

Other (not specified) 3 (<1) Tissue source

Cord blood unit 361 (65) Bone marrow 106 (19) Peripheral blood 55 (10) T-lymphocyte 17 (3) Placenta 6 (1) Adipose tissue 4 (1) Cardiac tissue 2 (<1) Pancreatic tissue 1 (<1) CD34 1 (<1) Missing 3 (<1)

Cell Type Infused Cord blood unit 358 (64) Unselected mononuclear cells 40 (7) Mesenchymal stromal cells 30 (5) T-Cells 43 (7) Stem Cells 36 (6) CD34 8 (1) Natural killer cells 10 (1) ALDH bright selected cells 7 (1) Adipose derived regenerative cells 4 (<1) Human umbilical cord perivascular 3 (<1)

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Characteristics N (%) Islet cells 1 (<1) Myeloid Progenitor Cells 2 (<1) Cardiosphere-derived cells (CAP-1002) 2 (<1) CD 19 – CAR T cells 1 (<1) Hematopoietic Progenitor Cells 1 (<1) Nucleated cells 1 (<1) Missing 7 (1)

*Several centers first collect certain data required for the completion Form 4000 at 1-year post infusion and cannot submit Form 4000 to the CIBMTR until this time. Cases continue to be registered for 2014 and 2015; data for these years are incomplete.

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Donor Cellular Infusion reported to CIBMTR on TED form for transplants between 2007 and 2016

Year of Transplant

Variable 2007‐2008 2009‐2010 2011‐2012 2013‐2014 2015‐2016*

Number of infusions 1551 1825 1734 1655 633

Number of patients 1524 1787 1692 1623 614

Number of centers 199 198 196 195 157

Indications

Planned 114 (7) 154 (9) 181 (11) 211 (13) 109 (18)

Treat disease 884 (58) 1044 (58) 902 (53) 859 (53) 253 (41)

Treat PTLD, EBV‐Lym 19 (1) 17 (<1) 19 (1) 16 (<1) 4 (<1)

Treat viral 22 (1) 38 (2) 50 (3) 54 (3) 29 (5)

Treat GVHD 131 (9) 188 (11) 165 (10) 153 (9) 61 (10)

Mixed Chimerism 123 (8) 142 (8) 158 (9) 158 (10) 72 (12)

Loss / Decreased Chimerism 45 (3) 67 (4) 76 (4) 61 (4) 27 (4)

Other 137 (9) 134 (7) 134 (8) 98 (6) 57 (9)

Missing 49 (3) 3 (<1) 7 (<1) 13 (<1) 2 (<1)

Cell Type

Lymphocytes 1231 (81) 1516 (85) 1443 (85) 1363 (84) 480 (78)

Fibroblasts 3 (<1) 14 (<1) 6 (<1) 3 (<1)

Dendritic cells 7 (<1) 4 (<1) 4 (<1) 4 (<1) 1 (<1)

Mesenchymal 35 (2) 78 (4) 86 (5) 60 (4) 21 (3)

Other cell type infused 542 (36) 476 (27) 314 (19) 440 (27) 206 (34) *Cases continue to be registered to CIBMTR for 2016

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TO: Autoimmune Diseases and Cellular Therapies Working Committee Members FROM: Marcelo C. Pasquini, MD, MS; Scientific Director for the Autoimmune Diseases and

Cellular Therapies Working Committee RE: Studies in Progress Summary CT10-01: Donor Leukocyte Infusion Versus Second Allogeneic Hematopoietic Stem Cell Transplantation for Disease Relapses after First Allogeneic Stem Cell Transplantation (N Frey/A Loren/D Porter) The study proposes to compare outcomes among patients with relapsed hematologic malignancies after allogeneic hematopoietic stem cell transplant who receive either a second allogeneic HCT, donor leukocyte infusion or no intervention. Factors associated with overall survival for DLI and second HCT are also of interest. Analysis report was done in Aug 2014 and was sent to the writing committee for comments. The study is in manuscript preparation now with the goal of submission. CT13-01: Utility of Unmanipulated Donor Lymphocyte Infusion (DLI) for the Treatment of Infections in Allogeneic Hematopoietic Cell Transplantation Recipients (G Akpek) The objective of this study is to describe clinical outcomes of DLI for treatment of infections. Protocol was presented at the statistical meeting in Sep 2014 and 109 eligible patients were registered with CIBMTR. This study has been in data collection since June of 2016, data collection has been a time consuming and challenging phase for this study. We are currently working with the last few centers on collecting there data 2 of whom are the largest contributing centers to the population. The goal is to have data collection complete by February of 2016 and to have this study in manuscript preparation by July 2017. The main barrier for this study was the data collection, which has been slow. In the future, with improvement of the cellular therapy forms, all data will be collected directly if little or no need for supplemental data collection. AC14-01: Long Term Outcomes after Autologous Hematopoietic Cell Transplantation for Rapidly Progressive Systemic Scleroderma (D Farge) This study is being done in collaboration with the Autoimmune Working Party from the EBMT. The objective of this study is to evaluate long term outcomes from autologous hematopoietic cell transplantation for rapidly progressive systemic sclerosis (SSc) from time of transplant to three and five years after transplantation. This study was presented at a December 2016 statistical meeting. The study is currently in data collection, and invitations are being sent out to centers requesting their participation in the study. The goal of the study is to have data collected and have the study in analysis by July 2017. AC16-01 Pattern of use and outcomes with donor lymphocyte infusion (DLI) after HLA-haploidentical allogeneic hematopoietic stem cell transplant (V Roy) The objective of this study is to describe the frequency of use of DLI and the efficacy and toxicity of DLI after HLA identical T-replete HCT. The study is currently in protocol development. The protocol was presented at the January 10th statistical meeting and the goal for this study is to be in analysis by July 2017. The approach for data collection here will be to utilize the newly developed CTED forms to obtain more details on the cellular therapy used after haploidentical donor HCT.

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A G E N D A CIBMTR WORKING COMMITTEE FOR AUTOIMMUNE ... · Reingold SC, Cohen J. Cell-Based...

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